What the trial was testing
The ROAR enrolled 206 patients with biliary tract cancer. The study was sponsored by Novartis Pharmaceuticals and tracked outcomes across the full group of patients who matched the trial's eligibility profile.
It was initial testing (phase 2). Trials at this stage are designed to produce evidence regulators and physicians can act on — not just observations to follow up later.
What the results showed
About 50% tumor response rate in BRAF-mutant biliary tract cancer.
The Lancet Oncology · 2020 · NCT02034110
These findings — that in advanced BRAF-mutant biliary tract cancer on dabrafenib plus trametinib — were published in the The Lancet Oncology and represent the headline result of the study.
Researchers tracked outcomes across 206 patients enrolled in the trial. The result was consistent enough across the group that the team felt confident reporting it.
What this means for patients
For patients with biliary tract cancer, this result changes the calculus on what to ask their care team about. Whether it changes day-to-day care depends on factors like disease subtype, prior treatments, and where the patient is in their care journey.
What you can do now
Dabrafenib (Tafinlar) plus trametinib (Mekinist) is FDA-approved for any solid tumor with a BRAF V600E mutation, including biliary tract cancer, and available now. Ask an oncologist about BRAF testing on your tumor — most centers test routinely for advanced bile duct cancer.
Eligibility for the treatments mentioned above depends on specific test results and clinical history. Bring this summary, the trial name, and your most recent labs or pathology report to your next visit.
Open biliary tract cancer trials
Ambispective Cohort Study of Precision Medicine for Primary Hepatobiliary Cancer Based on Next-generation Sequencing
Exploring the precise medicine of patients with primary hepatobiliary cancer. And evaluate the efficacy and safety of individualized treatment regimens for primary hepatobiliary cancer based on next-generation sequencing.
Pressurized Intraperitoneal Aerosolized Nab-Paclitaxel in Combination With Gemcitabine and Cisplatin for the Treatment of Biliary Tract Cancer Patients With Peritoneal Metastases
This phase I trial studies the side effects of pressurized intraperitoneal aerosolized chemotherapy (PIPAC) nab-paclitaxel in combination with gemcitabine and cisplatin in treating patients with biliary tract cancer that has spread to the peritoneum (peritoneal metastases). PIPAC involves the administration of intraperitoneal chemotherapy (anticancer drugs given directly to the lining of the abdomen). PIPAC uses a nebulizer (a device that turns liquids into a fine mist) which is connected to a high-pressure injector and inserted into the abdomen (part of the body that contains the digestive organs) during a laparoscopic procedure (a surgery using small incisions to introduce air and insert a camera and other instruments into the abdominal cavity for diagnosis and/or to perform routine surgical procedures). Pressurization of the liquid chemotherapy through the study device results in aerosolization (a fine mist or spray) of the chemotherapy intra-abdominally (into the abdomen), which results in the drug reaching more of the tissue as well as reaching deeper into the tissue, which reduces the amount of chemotherapy that needs to be used and potentially reduces side effect. Chemotherapy drugs, such as nab-paclitaxel, gemcitabine, and cisplatin, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving nab-paclitaxel via PIPAC in combination with standard of care gemcitabine and cisplatin may reduce side effects and make this chemotherapy regimen more tolerable in patients with biliary tract cancer that has spread to the spread to the peritoneum.